The collaboration will leverage the expertise of Queen’s University and Erytech’s ERYCAPS platform technology with the goal of generating in vivo proof-of-concept data in an arginase-1 deficiency animal model.
Arginase-1 deficiency is a rare, inherited disorder of the urea cycle caused by a mutation in the arginase-1 gene, resulting in the accumulation of toxic levels of the amino acid arginine in the blood. Symptoms generally appear in early infancy and include intellectual disability, non-ambulatory muscle stiffness and seizures. It is a debilitating, progressive disease with very limited treatment options currently available.
The collaboration between Erytech and Queen’s University aims to demonstrate the potential of Erytech’s eryminase to lower arginine in the inducible arginase-1 deficiency mouse model developed by the laboratory of Prof. Colin Funk of Queen’s University. Eryminase, a product candidate being developed by Erytech, consists of an arginine deiminase enzyme encapsulated in red blood cells using Erytech’s proprietary ERYCAPS platform technology. Erytech believes the encapsulation of the therapeutic enzymes in the red blood cells can provide effective, long-acting therapeutic activity with reduced toxicity.
Prof. Colin Funk, PhD, Queen’s University, commented, “Arginase-1 deficiency is a severe, rare disorder affecting a biochemical pathway that disposes of toxic ammonia. Normally, our bodies are very efficient at removing any ammonia that accumulates after eating a protein-rich meal. However, in patients with arginase-1 deficiency, the ammonia is 'partially detoxified' leading to a large accumulation of the amino acid arginine in the patient’s blood and brain.
“ERYTECH’s product candidate eryminase aims to reduce the level of arginine in blood and thus, has significant potential to reduce negative consequences of this disorder. We look forward to working with ERYTECH to advance their preclinical program.”
Dr. Alexander Scheer, PhD, Chief Scientific Officer of Erytech, added, “This is our second collaboration in the field of rare metabolic diseases that underscores the scope of our platform and its applicability to highly specialized and rare conditions beyond oncology. We are very pleased to enter this collaboration with Queen’s University and look forward to working closely on this important program with Dr. Funk who specializes in research related to urea cycle disorders.”